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Congenital, familial and genetic disorders – page 44

  • 803 clinical trials
  • 311 recruiting
  • 375 conditions
  • 27 countries

Conditions in this area

375 conditions with trials, the busiest first

Duchenne muscular dystrophy28Factor VIII deficiency25Sickle cell disease25Cystic fibrosis24Hereditary angioedema22Hypertrophic cardiomyopathy17Spinal muscular atrophy17Type IIa hyperlipidaemia15Glycogen storage disease type II14Factor IX deficiency13Huntington's disease13Sickle cell anaemia13Congenital cystic kidney disease12Myotonic dystrophy12Non-small cell lung cancer12Osteochondrodysplasia12Adrenogenital syndrome11Alpha-1 antitrypsin deficiency11K-ras gene mutation11Retinitis pigmentosa11Severe myoclonic epilepsy of infancy11Thalassaemia11Epidermolysis bullosa10Fabry's disease10+351

Clinical trials in Congenital, familial and genetic disorders

803 trials across Europe, recruiting first

Type of trial
Recruitment status
Quick filters

803 clinical trials

K-ras gene mutation+3

Not recruiting

Study of JDQ443, TNO155, and Tislelizumab for Patients with Advanced Solid Tumors with KRAS G12C Mutation

InvestigationalNo placebo
Countries:BelgiumBelgium
  • Participants:18–64 years · 65+ years
  • Substances:Batoprotafib
  • Sponsor:Novartis Pharma AG

Ornithine transcarbamoylase deficiency

Not recruiting

Study on ECUR-506A and ECUR-506D for Male Infants Under 9 Months with Neonatal Onset Ornithine Transcarbamylase Deficiency

InvestigationalNo placebo
Countries:SpainSpain
  • Participants:0–17 years
  • Substances:Adeno-Associated Virus Serotype Rh79 Containing The Human Otc Gene
  • Sponsor:Iecure Inc.

Tuberous sclerosis complex

Not recruiting

Study on Basimglurant for Children, Adolescents, and Young Adults with Tuberous Sclerosis Complex-Related Seizures

Investigational
Countries:ItalyItaly
  • Participants:0–17 years · 18–64 years
  • Substances:Basimglurant
  • Sponsor:Noema Pharma AG

Duchenne muscular dystrophy

Not recruiting

Study on the Effects of Satralizumab in Children and Adolescents with Duchenne Muscular Dystrophy

InvestigationalNo placebo
Countries:DenmarkDenmark
  • Participants:0–17 years
  • Sponsor:F. Hoffmann-La Roche AG

Hepato-lenticular degeneration

Not recruiting

Study of Trientine Dihydrochloride (Cufence) to Evaluate Its Effects and Safety in Patients with Wilson's Disease

Registered drugNo placebo
Countries:DenmarkDenmark
  • Participants:0–17 years · 18–64 years · 65+ years
  • Substances:Trientine Dihydrochloride
  • Sponsor:Univar Solutions B.V.

Hormone receptor positive HER2 negative breast cancer+1

Not recruiting

Study on Alpelisib and Fulvestrant for Advanced Breast Cancer in Patients with PIK3CA Mutation and Hormone-Receptor Positive, HER2 Negative Tumors

Registered drugNo placebo
Countries:The NetherlandsThe Netherlands
  • Participants:18–64 years · 65+ years
  • Substances:Alpelisib
  • Sponsor:BOOG Study Center B.V.

Polycythaemia vera+10

Not recruiting

Study on Long-Term Safety of Ruxolitinib, Panobinostat, and Siremadlin for Patients Continuing Treatment from Previous Studies

Registered drugNo placebo
Countries:GermanyGermany
  • Participants:0–17 years · 18–64 years · 65+ years
  • Substances:Panobinostat
  • Sponsor:Novartis Pharma Services AG

Congenital nephrogenic diabetes insipidus

Not recruiting

Study of Fluconazole Treatment for Patients with Congenital Nephrogenic Diabetes Insipidus

Registered drugNo placebo
Countries:DenmarkDenmark
  • Participants:18–64 years · 65+ years
  • Substances:Clotrimazole
  • Sponsor:Odense University Hospital

Usher's syndrome

Not recruiting

Study on Long-Term Safety and Effects of SAR421869 for Patients with Usher Syndrome Type 1B

InvestigationalNo placebo
Countries:FranceFrance
  • Participants:18–64 years
  • Substances:Lentiviral Vector Derived From The Equine Infectious Anaemia Virus Containing The Human Myo7A Gene
  • Sponsor:Sanofi-Aventis Recherche & Developpement

Thalassaemia beta

Not recruiting

Long-Term Safety and Efficacy Study of Betibeglogene Autotemcel Gene Therapy for Patients with Transfusion-Dependent Beta-Thalassemia

InvestigationalNo placebo
Countries:FranceFrance
  • Participants:0–17 years · 18–64 years
  • Substances:Betibeglogene Autotemcel
  • Sponsor:Genetix Biotherapeutics Inc.

Aicardi-Goutieres syndrome

Not recruiting

Study on the Safety of Censavudine (TPN-101) for Patients with Aicardi-Goutières Syndrome

InvestigationalNo placebo
Countries:FranceFrance
  • Participants:0–17 years · 18–64 years
  • Substances:Censavudine
  • Sponsor:Transposon Therapeutics Inc.

Fabry's disease

Not recruiting

Study on the Long-Term Safety and Effectiveness of Pegunigalsidase Alfa for Adults with Fabry Disease

InvestigationalNo placebo
Countries:BelgiumBelgium
  • Participants:18–64 years
  • Substances:Pegunigalsidase Alfa
  • Sponsor:Chiesi Farmaceutici S.p.A.

Substances studied in this area

The therapies tested most often in Congenital, familial and genetic disorders trials.

Sirolimus21 clinical trialsSodium Chloride21 clinical trialsPrednisolone12 clinical trialsPlerixafor11 clinical trialsBusulfan10 clinical trialsIvacaftor9 clinical trialsMitapivat9 clinical trialsParacetamol8 clinical trialsTezacaftor8 clinical trials
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